Skip to main content
    • Aa
    • Aa

Emerging strategies for cell and gene therapy of the muscular dystrophies

  • Lindsey A. Muir (a1) and Jeffrey S. Chamberlain (a2)

The muscular dystrophies are a heterogeneous group of over 40 disorders that are characterised by muscle weakness and wasting. The most common are Duchenne muscular dystrophy and Becker muscular dystrophy, which result from mutations within the gene encoding dystrophin; myotonic dystrophy type 1, which results from an expanded trinucleotide repeat in the myotonic dystrophy protein kinase gene; and facioscapulohumeral dystrophy, which is associated with contractions in the subtelomeric region of human chromosome 1. Currently the only treatments involve clinical management of symptoms, although several promising experimental strategies are emerging. These include gene therapy using adeno-associated viral, lentiviral and adenoviral vectors and nonviral vectors, such as plasmid DNA. Exon-skipping and cell-based therapies have also shown promise in the effective treatment and regeneration of dystrophic muscle. The availability of numerous animal models for Duchenne muscular dystrophy has enabled extensive testing of a wide range of therapeutic approaches for this type of disorder. Consequently, we focus here on the therapeutic developments for Duchenne muscular dystrophy as a model of the types of approaches being considered for various types of dystrophy. We discuss the advantages and limitations of each therapeutic strategy, as well as prospects and recent successes in the context of future clinical applications.

Corresponding author
*Corresponding author: Jeffrey S. Chamberlain, Department of Neurology, University of Washington School of Medicine, HSB Room K233, Box 357720, Seattle, Washington 98195, USA. Tel: +1 206 616 6645; Fax: +1 206 616 8272; E-mail:
Linked references
Hide All

This list contains references from the content that can be linked to their source. For a full set of references and notes please see the PDF or HTML where available.

4 R. Tawil (1998) Facioscapulohumeral dystrophy: A distinct regional myopathy with a novel molecular pathogenesis. Annals of Neurology 43, 279-282

5 D. Bansal and K.P. Campbell (2004) Dysferlin and the plasma membrane repair in muscular dystrophy. Trends in Cell Biology 14, 206-213

6 K.E. Davies and K.J. Nowak (2006) Molecular mechanisms of muscular dystrophies: old and new players. Nature Reviews Molecular Cell Biology 7, 762-773

7 A.P. Monaco (1988) An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 2, 90-95

9 X. Huang (2000) Structure of a WW domain containing fragment of dystrophin in complex with beta-dystroglycan. Nature Structural and Molecular Biology 7, 634-638

11 J.M. Ervasti and K.P. Campbell (1993) A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin. Journal of Cell Biology 122, 809-823

13 P.R. Turner (1988) Increased protein degradation results from elevated free calcium levels found in muscle from mdx mice. Nature 335, 735-738

14 C.L. Batchelor and S.J. Winder (2006) Sparks, signals and shock absorbers: how dystrophin loss causes muscular dystrophy. Trends in Cell Biology 16, 198-205

15 N.P. Whitehead (2006) Streptomycin reduces stretch-induced membrane permeability in muscles from mdx mice. Neuromuscular Disorders 16, 845-854

16 L.L. Baumbach (1989) Molecular and clinical correlations of deletions leading to Duchenne and Becker muscular dystrophies. Neurology 39, 465-474

17 E. Ozawa (1998) From dystrophinopathy to sarcoglycanopathy: Evolution of a concept of muscular dystrophy. Muscle & Nerve 21, 421-438

18 K.P. Campbell (1995) Three muscular dystrophies: Loss of cytoskeleton-extracellular matrix linkage. Cell 80, 675-679

21 G.B. Banks , J.S. Chamberlain and R.S. Krauss (2008) Chapter 9, The value of mammalian models for Duchenne muscular dystrophy in developing therapeutic strategies. Current Topics in Developmental Biology 84, 431-453

23 J.S. Chamberlain (2002) Gene therapy of muscular dystrophy. Human Molecular Genetics 11, 2355-2362

24 S.B. England (1990) Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 343, 180-182

25 K. Matsumura (1994) Immunohistochemical analysis of dystrophin-associated proteins in Becker/Duchenne muscular dystrophy with huge in-frame deletions in the NH2-terminal and rod domains of dystrophin. Journal of Clinical Investigation 93, 99-105

26 S.Q. Harper (2002) Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nature Medicine 8, 253-261

27 S.F. Phelps (1995) Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Human Molecular Genetics 4, 1251-1258

28 M. Sakamoto (2002) Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochemical and Biophysical Research Communications 293, 1265-1272

30 M. Ishikawa-Sakurai (2004) ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to {beta}-dystroglycan. Human Molecular Genetics 13, 693-702

33 S.C. Gilchrist (2002) Immune response to full-length dystrophin delivered to DMD muscle by a high-capacity adenoviral vector. Molecular Therapy 6, 359-368

35 J.M. Tinsley (1992) Primary structure of dystrophin-related protein. Nature 360, 591-593

36 K. Matsumura (1992) Association of dystrophin-related protein with dystrophin-associated proteins in mdx mouse muscle. Nature 360, 588-591

39 T.S. Khurana (1991) Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle. Neuromuscular Disorders 1, 185-194

40 Y. Mizuno (1993) Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD-carriers and control subjects. Journal of the Neurological Sciences 119, 43-52

41 A.P. Weir , J.E. Morgan and K.E. Davies (2004) A-utrophin up-regulation in mdx skeletal muscle is independent of regeneration. Neuromuscular Disorders 14, 19-23

42 J.R. Deol (2007) Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin. Molecular Therapy 15, 1767-1774

43 J.M. Tinsley (1996) Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349-353

44 A.E. Deconinck (1997) Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy. Cell 90, 717-727

45 M. Cerletti (2003) Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Therapy 10, 750-757

46 G.L. Odom (2008) Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Molecular Therapy 16, 1539-1545

50 P. Gregorevic (2004) Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nature Medicine 10, 828-834

51 G. Gao (2004) Clades of adeno-associated viruses are widely disseminated in human tissues. Journal of Virology 78, 6381-6388

52 H. Chao (2000) Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Molecular Therapy 2, 619-623

53 D. Duan (2001) Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation. Journal of Virology 75, 7662-7671

54 D. Grimm (2003) Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 102, 2412-2419

55 C.L. Halbert , J.M. Allen and A.D. Miller (2001) Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. Journal of Virology 75, 6615-6624

57 B.C. Schnepp (2009) Infectious molecular clones of adeno-associated virus isolated directly from human tissues. Journal of Virology 83, 1456-1464

58 J.R. Chamberlain (2004) Gene targeting in stem cells from individuals with osteogenesis imperfecta. Science 303, 1198-1201

59 K. Inagaki (2007) DNA palindromes with a modest arm length of greater than or equal to 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice. Journal of Virology 81, 11290-11303

61 C.S. Manno (2003) AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972

62 K. Yuasa (2002) Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Therapy 9, 1576-1588

63 D. Hartigan-O'Connor (2001) Immune evasion by muscle-specific gene expression in dystrophic muscle. Molecular Therapy 4, 525-533

65 C.S. Manno (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Medicine 12, 342-347

66 F. Mingozzi and K. High (2007) Immune responses to AAV in clinical trials. Current Gene Therapy 7, 316-324

67 F. Mingozzi (2007) CD8+ T-cell responses to adeno-associated virus capsid in humans. Nature Medicine 13, 419-422

68 H. Jiang (2006) Effects of transient immunosuppression on adeno-associated virus-mediated liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108, 3321-3328

69 D. Townsend (2008) Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Molecular Therapy 16, 832-835

70 C. Mah (2005) Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors. Gene Therapy 12, 1405-1409

71 Z. Wang (2005) Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nature Biotechnology 23, 321-328

72 K. Inagaki (2006) Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Molecular Therapy 14, 45-53

73 Y. Yue (2008) A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Molecular Therapy 16, 1944-1952

74 B.R. Schultz and J.S. Chamberlain (2008) Recombinant adeno-associated virus transduction and integration. Molecular Therapy 16, 1189-1199

75 G.B. Banks (2007) Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain. Human Molecular Genetics 16, 2105-2113

76 K.E. Wells (2003) Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle. Neuromuscular Disorders 13, 21-31

77 L.M. Judge , M. Haraguchi and J.S. Chamberlain (2006) Dissecting the signaling and mechanical functions of the dystrophin-glycoprotein complex. Journal of Cell Science 119, 1537-1546

78 Y. Lai (2009) Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. Journal of Clinical Investigation 119, 624-635

80 Y.M. Kobayashi (2008) Sarcolemma-localized nNOS is required to maintain activity after mild exercise. Nature 456, 511-515

81 G.B. Banks (2008) Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin. Human Molecular Genetics 17, 3975-3986

82 G.B. Banks , J.S. Chamberlain and S.C. Froehner (2009) Truncated dystrophins can influence neuromuscular synapse structure. Molecular and Cellular Neuroscience 40, 433-441

86 D. Duan , Y. Yue and J.F. Engelhardt (2001) Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison. Molecular Therapy 4, 383-391

87 Y. Lai (2006) Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors. Human Gene Therapy 17, 1036-1042

88 C.L. Halbert , J.M. Allen and A.D. Miller (2002) Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nature Biotechnology 20, 697-701

89 A. Ghosh (2007) A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner. Molecular Therapy 16, 124-130

90 S. Li (2005) Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin. Gene Therapy 12, 1099-1108

91 S. Hacein-Bey-Abina (2003) A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. New England Journal of Medicine 348, 255-256

92 A. Ciuffi (2006) Integration site selection by HIV-based vectors in dividing and growth-arrested IMR-90 lung fibroblasts. Molecular Therapy 13, 366-373

93 B.C. Beard (2007) Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus. Human Gene Therapy 18, 423-434

94 J. Barquinero , H. Eixarch and M. Perez-Melgosa Retroviral vectors: new applications for an old tool. Gene Therapy 11, S3-S9

95 T. Kafri (1997) Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nature Genetics 17, 314-317

96 D. Bonci (2003) ‘Advanced’ generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Therapy 10, 630-636

98 A. Annoni (2007) The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4 + CD25+ regulatory T cells. Blood 110, 1788-1796

99 E. Bachrach (2006) Muscle engraftment of myogenic progenitor cells following intraarterial transplantation. Muscle & Nerve 34, 44-52

100 A. Dellavalle (2007) Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells. Nature Cell Biology 9, 255-267

101 R. Kumar-Singh and J.S. Chamberlain (1996) Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Human Molecular Genetics 5, 913-921

104 D. Hartigan-O'Connor (2002) Generation and growth of adenoviral vectors. Methods in Enzymology 346, 224-246

106 R. Gilbert (2003) Prolonged dystrophin expression and functional correction of mdx mouse muscle following gene transfer with a helper-dependent (gutted) adenovirus-encoding murine dystrophin. Human Molecular Genetics 12, 1287-1299

107 L.T. Su (2005) Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector. Circulation 112, 1780-1788

109 G. Acsadi (1994) A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Human Molecular Genetics 3, 579-584

110 P.W. Zoltick (2001) Biology of E1-deleted adenovirus vectors in nonhuman primate muscle. Journal of Virology 75, 5222-5229

111 D.A. Muruve (1999) Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Human Gene Therapy 10, 965-976

112 N. Brunetti-Pierri (2004) Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Human Gene Therapy 15, 35-46

113 S.E. Raper (2003) Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Molecular Genetics and Metabolism 80, 148-158

115 J. Wolff (1990) Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468

116 G. Zhang (2004) Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of Duchenne muscular dystrophy. Human Gene Therapy 15, 770-782

117 J.E. Hagstrom (2004) A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Molecular Therapy 10, 386-398

118 P. Richard (2005) Amphiphilic block copolymers promote gene delivery in vivo to pathological skeletal muscles. Human Gene Therapy 16, 1318-1324

119 P. Chollet (2002) Side-effects of a systemic injection of linear polyethylenimine-DNA complexes. The Journal of Gene Medicine 4, 84-91

120 V.S. Trubetskoy (2003) Recharging cationic DNA complexes with highly charged polyanions for in vitro and in vivo gene delivery. Gene Therapy 10, 261-271

121 N.B. Romero (2004) Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Human Gene Therapy 15, 1065-1076

125 Y. Takeshima (1995) Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe. Journal of Clinical Investigation 95, 515-520

126 Z.A. Pramono (1996) Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence. Biochemical and Biophysical Research Communications 226, 445-449

127 S. Shibahara (1989) Inhibition of human immunodeficiency virus (HIV-1) replication by synthetic oligo-RNA derivatives. Nucleic Acids Research 17, 239-252

128 M.G. Dunckley (1998) Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Human Molecular Genetics 7, 1083-1090

129 G. McClorey (2006) Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Therapy 13, 1373-1381

130 J.C. van Deutekom (2001) Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Human Molecular Genetics 10, 1547-1554

132 J. Summerton and D. Weller (1997) Morpholino antisense oligomers: design, preparation, and properties. Antisense and Nucleic Acid Drug Development 7, 187-195

133 T. Yokota (2009) A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Archives of Neurology 66, 32-38

134 P. Sazani (2002) Systemically delivered antisense oligomers upregulate gene expression in mouse tissues. Nature Biotechnology 20, 1228-1233

135 B. Lebleu (2008) Cell penetrating peptide conjugates of steric block oligonucleotides. Advanced Drug Delivery Reviews 60, 517-529

136 B. Wu (2009) Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Molecular Therapy 17, 864-871

137 G.D. Ivanova (2008) Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Research 36, 6418-6428

138 J. Alter (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nature Medicine 12, 175-177

139 S. Fletcher (2007) Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Molecular Therapy 15, 1587-1592

141 N. Jearawiriyapaisarn (2008) Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Molecular Therapy 16, 1624-1629

143 L.J. Popplewell (2009) Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Molecular Therapy 17, 554-561

144 C. Mitrpant (2009) By-passing the nonsense mutation in the 4 (CV) mouse model of muscular dystrophy by induced exon skipping. The Journal of Gene Medicine 11, 46-56

146 A. Goyenvalle (2004) Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping. Science 306, 1796-1799

147 M.A. Denti (2006) Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice. Human Gene Therapy 17, 565-574

148 H.A. Heemskerk (2009) In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. The Journal of Gene Medicine 11, 257-266

149 D. Skuk and J.P. Tremblay (2003) Myoblast transplantation: the current status of a potential therapeutic tool for myopathies. Journal of Muscle Research and Cell Motility 24, 285-300

150 D. Skuk (2007) First test of a “high-density injection” protocol for myogenic cell transplantation throughout large volumes of muscles in a Duchenne muscular dystrophy patient: eighteen months follow-up. Neuromuscular Disorders 17, 38-46

151 D. Skuk (2007) Ischemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategies. Transplantation 84, 1307-1315

152 D. Montarras (2005) Direct Isolation of Satellite Cells for Skeletal Muscle Regeneration. Science 309, 2064-2067

153 B. Peault (2007) Stem and progenitor cells in skeletal muscle development, maintenance, and therapy. Molecular Therapy 15, 867-877

154 C. Webster and H. Blau (1990) Accelerated age-related decline in replicative life-span of Duchenne muscular dystrophy myoblasts: implications for cell and gene therapy. Somatic Cell and Molecular Genetics 16, 557-565

155 Y. Fan (1996) Rapid death of injected myoblasts in myoblast transfer therapy. Muscle & Nerve 19, 853-860

156 J.P. Tremblay and D. Skuk (2008) Another new “super muscle stem cell” leaves unaddressed the real problems of cell therapy for Duchenne muscular dystrophy. Molecular Therapy 16, 1907-1909

157 B.M. Deasy (2002) Mechanisms of muscle stem cell expansion with cytokines. Stem Cells 20, 50-60

158 C.A. Collins (2005) Stem cell function, self-renewal, and behavioral heterogeneity of cells from the adult muscle satellite cell niche. Cell 122, 289-301

159 D.D.W. Cornelison (2008) Context matters: In vivo and in vitro influences on muscle satellite cell activity. Journal of Cellular Biochemistry 105, 663-669

160 E. Gussoni (1999) Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature 401, 390-394

161 J.Y. Lee (2000) Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing. Journal of Cell Biology 150, 1085-1100

162 Y. Torrente (2007) Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients. Cell Transplantation 16, 563-577

163 G. Ferrari (1998) Muscle regeneration by bone marrow-derived myogenic progenitors. Science 279, 1528-1530

164 M. Dezawa (2005) Bone marrow stromal cells generate muscle cells and repair muscle degeneration. Science 309, 314-317

165 M. Sampaolesi (2003) Cell therapy of {alpha}-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science 301, 487-492

166 M. Sampaolesi (2006) Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature 444, 574-579

167 T. Barberi (2007) Derivation of engraftable skeletal myoblasts from human embryonic stem cells. Nature Medicine 13, 642-648

168 R. Darabi (2008) Functional skeletal muscle regeneration from differentiating embryonic stem cells. Nature Medicine 14, 134-143

169 E. Kimura (2008) Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy. Human Molecular Genetics 17, 2507-2517

170 K. Takahashi (2007) Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861-872

171 J. Yu (2007) Induced pluripotent stem cell lines derived from human somatic cells. Science 318, 1917-1920

172 P. Rebuzzini (2008) Chromosome number variation in three mouse embryonic stem cell lines during culture. Cytotechnology 58, 17-23

173 P. Catalina (2008) Human ESCs predisposition to karyotypic instability: Is a matter of culture adaptation or differential vulnerability among hESC lines due to inherent properties? Molecular Cancer 7, 76

174 J. Hanna (2007) Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 318, 1920-1923

175 G. Dickson (2002) Recombinant micro-genes and dystrophin viral vectors. Neuromuscular Disorders 12, S40-S44

176 M.A.F.V. Goncalves (2008) Targeted chromosomal insertion of large DNA into the human genome by a fiber-modified high-capacity adenovirus-based vector system. PLoS ONE 3, 3084

177 M.Z. Salva (2007) Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Molecular Therapy 15, 320-329

178 A. Zaldumbide and R.C. Hoeben (2007) How not to be seen: immune-evasion strategies in gene therapy. Gene Therapy 15, 239-246

179 J.C. van Deutekom (2007) Local dystrophin restoration with antisense oligonucleotide PRO051. New England Journal of Medicine 357, 2677-2686

180 S.E. Newey (2000) Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex. Current Biology 10, 1295-1298

181 M.E. Adams , H.A. Mueller and S.C. Froehner (2001) In vivo requirement of the {alpha}-syntrophin PDZ domain for the sarcolemmal localization of nNOS and aquaporin-4. Journal of Cell Biology 155, 113-122

D.J. Blake (2002) Function and genetics of dystrophin and dystrophin-related proteins in muscle. Physiological Reviews 82, 291-329

G. L. Odom , P. Gregorevic and J. S. Chamberlain (2007) Viral-mediated gene therapy for the muscular dystrophies: Successes, limitations and recent advances. Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 1772, 243-262

Recommend this journal

Email your librarian or administrator to recommend adding this journal to your organisation's collection.

Expert Reviews in Molecular Medicine
  • ISSN: -
  • EISSN: 1462-3994
  • URL: /core/journals/expert-reviews-in-molecular-medicine
Please enter your name
Please enter a valid email address
Who would you like to send this to? *


Full text views

Total number of HTML views: 1
Total number of PDF views: 11 *
Loading metrics...

Abstract views

Total abstract views: 142 *
Loading metrics...

* Views captured on Cambridge Core between September 2016 - 30th May 2017. This data will be updated every 24 hours.